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Alnylam Development Pipeline

Alnylam’s pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs):

  • Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases;
  • Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and,
  • Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases.

Development Programs
Discovery
Development
Phase 1
Phase 2
Phase 3
GENETIC MEDICINES
Patisiran (ALN-TTR02)
Revusiran (ALN-TTRsc)
ALN-AT3
ALN-CC5
ALN-AS1
ALN-AAT
ALN-GO1
ALN-TMP
CARDIO-METABOLIC DISEASES
ALN-PCSsc
ALN-AC3
ALN-ANG
ALN-AGT
HEPATIC INFECTIOUS DISEASES
ALN-HBV
ALN-HDV
ALN-PDL
Discovery
Development
Phase 1
Phase 2
Phase 3



GENETIC MEDICINES

TTR-Mediated Amyloidosis
  - Patisiran (ALN-TTR02)
  - Revusiran (ALN-TTRsc)

Hemophilia and Rare Bleeding Disorders - ALN-AT3

Complement-Mediated Diseases - ALN-CC5

Hepatic Porphyrias - ALN-AS1

Alpha-1 Antitrypsin Deficiency - ALN-AAT

Primary Hyperoxaluria - ALN-GO1

Beta-Thalassemia / Iron Overload Disorders - ALN-TMP

Additional Genetic Medicine Programs

CARDIO-METABOLIC DISEASES

Hypercholesterolemia ALN-PCSsc

Mixed Hyperlipidemia and Hypertriglyceridemia - ALN-ANG

Hypertriglyceridemia - ALN-AC3

Hypertension / Preeclampsia - ALN-AGT

Additional Cardio-Metabolic Programs

HEPATIC INFECTIOUS DISEASES

Hepatitis B Virus Infection - ALN-HBV

Hepatitis D Virus Infection - ALN-HDV

Chronic Liver Infection - ALN-PDL

Additional Hepatic ID Programs